CD133

SUNNYVALE, Calif., Sept. 06, 2023 (GLOBE NEWSWIRE) -- BioCardia®, Inc. [Nasdaq: BCDA], a developer of cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary disease, today announced a clarification and next steps on its autologous CardiAMP cell therapy programs. Based on the recent interim results in the CardiAMP autologous cell therapy for the treatment of heart failure (BCDA-01), the Company is exploring development of a new Phase III clinical trial protocol. The Finkelstein Schoenfeld (FS) primary composite endpoint used in the CardiAMP HF Trial has tiers of outcomes in decreasing order of importance: heart death equivalent, major adverse cardiac and cerebrovascular MACCE, and Six Minute Walk Distance (6MWD). Since the primary FS endpoint is a composite of these elements, meeting the FS endpoint may be met even if one or more of the components does not individually demonstrate statistical significance. This has implications for the future trial design. The CardiAMP HF Trial interim results showed that the first two most important tier outcomes, occurring in 30% of the study patients, could have contributed sufficiently toward efficacy given a longer follow-up period than one year. If these interim results are replicated in a future study in which the third tier 6MWD is replaced with a more objective endpoint, there may be a pathway to a successful trial for product registration in the United States. The FDA has previously expressed a preference for replacing the 6MWD with Cardiopulmonary Exercise Testing, which is a more objective outcome. We are exploring with the CardiAMP HF Study Executive Steering Committee whether to fine tune eligibility criteria for patients based on current data and to replace the 6MWD with Cardiopulmonary Exercise Testing or another other more objective third-tier outcome measurement. Such a protocol may also be capital efficient by eliminating measures that have already been gathered in the ongoing CardiAMP HF study while continuing to utilize the CMS reimbursement program currently in place. Using the cell population analysis screening criterion to set patient dosage, rather than using it to exclude patients from the study, could accelerate enrollment. CardiAMP in Chronic Myocardial Ischemia, or BCDA-02, has a generally accepted mechanism of action of microvascular revascularization driven by the CD34 and CD133 components of the dosage. It is a different indication from the heart failure indication and members of the Steering Committee believe it has great potential to be successful even if the heart failure indication is not ultimately successful. Microvascular revascularization and repair to reduce pain and enhance heart performance in a relatively healthy heart is viewed as an easier challenge for the cells to overcome than helping a heart already in failure to recover. This study is on track to complete the roll in cohort enrollment in Q4 and advance to its randomized double blind pivotal trial. BioCardia expects to focus its resources on accelerating this pivotal program ahead and will be incorporating strategies to enhance enrollment. “We have three synergistic clinical programs for the treatment of ischemic heart disease utilizing the leading transendocardial delivery platform which we also developed,” continued Peter Altman. “Our expectation is that we can move all of these programs through significant milestones in the next year on less capital than we have utilized in previous years.” Anticipated Upcoming Milestones and Events: BCDA-01: CardiAMP Cell Therapy for Heart Failure Phase III Trial Q3 2023: Clarity on Second Pivotal Study DesignQ4 2023: Japan PMDA Formal Consultation BCDA-02: CardiAMP Cell Therapy for Chronic Myocardial Ischemia Phase III Trial Q4 2023: Completion of Roll-in Cohort and Transition to Randomized Pivotal Trial BCDA-03: NK1R+ MSC Allogeneic Cell Therapy in ischemic HFrEF Phase I/II Trial Q3 2023: First Patient Enrolled Helix Biotherapeutic Delivery System Q4 2023: Completion of Enrollment in Partner CellProthera’s EXCELLENT TrialQ4 2023: Update on Licensing / Partnerships About BioCardia® BioCardia, Inc., headquartered in Sunnyvale, California, is developing cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary disease. CardiAMP™ autologous and NK1R+ allogeneic cell therapies are the Company’s biotherapeutic platforms that enable four product candidates in development. The CardiAMP Cell Therapy Heart Failure Trial investigational product has been granted Breakthrough designation by the FDA, has CMS reimbursement, and is supported financially by the Maryland Stem Cell Research Fund. The CardiAMP Chronic Myocardial Ischemia Trial also has CMS Reimbursement. BioCardia also partners with other biotherapeutic companies to provide its delivery systems and development support to their programs studying therapies for the treatment of heart failure, chronic myocardial ischemia and acute myocardial infarction. For more information visit: www.BioCardia.com. Forward Looking Statements: This press release contains forward-looking statements that are subject to many risks and uncertainties. Forward-looking statements include, among other things, statements relating to future data analysis, future protocol submissions to FDA, anticipated milestones and events, conclusions of results based on interim data generated by the CardiAMP Heart Failure statistics core, the likelihood of safety and patient benefit, and the ultimate success of our clinical cell therapy programs. These forward-looking statements are made as of the date of this press release. We may use terms such as “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “approximately” or other words that convey the uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained herein, we caution you that forward-looking statements are not guarantees of future performance and that our actual results may differ materially from the forward-looking statements contained in this press release. Factors that could cause or contribute to such differences include, but are not limited to, the Company’s liquidity position and its ability to raise additional funds, as well as the Company’s ability to successfully progress its clinical trials. As a result of these factors, we cannot assure you that the forward-looking statements in this press release will prove to be accurate. Additional factors that could materially affect actual results can be found in BioCardia’s Form 10-K filed with the Securities and Exchange Commission on March 29, 2023, under the caption titled “Risk Factors” and in its subsequently filed Quarterly Reports on Form 10-Q. BioCardia expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law. Media Contact:Miranda Peto, Marketing / Investor RelationsEmail: mpeto@BioCardia.comPhone: 650-226-0120 Investor Contact:David McClung, Chief Financial OfficerEmail: investors@BioCardia.comPhone: 650-226-0120

Elevation Oncology's workforce shake-up didn’t stop at the boardroom door, with CEO Shawn Leland also departing. If last year was marred by continual stories of biotechs laying off staff and pruning back their pipelines to survive, then this week’s news suggests the new year hasn’t quite brought the fresh start the industry was hoping for. In a postmarket announcement yesterday, cell-therapy-focused Century Therapeutics announced it is de-prioritizing investment in CNTY-103, a CD133-targeting, chimeric antigen receptor, iPSC-derived NK (CAR-iNK) cell therapy candidate in preclinical development for glioblastoma. A discovery program for hematologic malignancies will also be discarded. Instead, the biotech will focus on its lead product CNTY-101, a CD19-targeting allogeneic cell therapy in phase 1 trials for relapsed/refractory B-cell lymphoma as well as a pair of gamma delta T-cell therapy candidates in preclinical development for B-cell malignancies and solid tumors. The reprioritization will allow the company to close its lab operations in Seattle and Hamilton, with research work consolidated in Philadelphia. While it means a quarter of the company’s workforce will be heading to the exits, the biotech explained that this will give it a cash runway of at least three years. The staffing shake-up even extended to the top ranks of the company, with R&D chief Hy Levitsky, M.D., Ph.D., resigning effective the end of the month. “As our confidence in the disruptive potential of our technology platform and prioritized pipeline programs continues to increase, we have implemented these cost-saving measures to rightsize the organization and further extend our cash runway to enable achievement of key milestones,” CEO Lalo Flores, Ph.D., said in a release. “As a result, we are losing many valued colleagues, which is an incredibly difficult decision, and we would like to thank each of them for their contributions.” It was a similar story at Elevation Oncology, which announced this morning that it was pausing investment in seribantumab. The fully human IgG2 monoclonal antibody, which is designed to bind to human epidermal growth factor receptor 3, had been on course to deliver further interim data from its phase 2 study in patients with solid tumors harboring NRG1 gene fusions. The company touted initial findings from the trial at the 2022 American Society of Clinical Oncology annual meeting as showing positive clinical proof-of-concept data. But now, the biotech has shelved the antibody unless or until a partner is found. Elevation’s focus will be on EO-3021, an antibody-drug conjugate targeting Claudin18.2 that’s entered phase 1 trials for solid tumors. Elevation acquired the ex-China rights to the drug from CSPC Megalith Biopharmaceutical for $27 million in July. Unfortunately, it’s a journey that 30% of Elevation’s existing workforce will no longer be required for. Again, the workforce shake-up didn’t stop at the boardroom door, with CEO Shawn Leland also departing. The situation was terminal for Nabriva Therapeutics, which has now shut shop entirely. The Irish biotech had already resorted to laying off 40% of staff and suspending its early-stage R&D projects in November. But the commercial refocus wasn’t enough to stay afloat, and the company announced this morning that it’s winding down operations as it looks for a buyer for its marketed assets like the antibiotic Xenleta. This latest spate of news caps off a couple of days that also saw Fate Therapeutics announce a raft of changes including layoffs, pipeline culls and ending a Johnson & Johnson collaboration. Meanwhile, Graphite Bio threatened “operational efficiencies to extend its cash position” after the first patient dosed with its lead gene therapy candidate experienced a serious adverse event.

Uni-Rare Study will improve clinical understanding of more IRDs and boost development of potential therapies. COLUMBIA, Md., Nov. 10, 2022 /PRNewswire/ -- The Foundation Fighting Blindness, the world's leading organization committed to finding treatments and cures for blinding retinal diseases, is committing at least $8.6 million for its Uni-Rare Study, a new natural history study for approximately 1,500 people with one of more than 300 rare genes associated with inherited retinal diseases (IRDs) including: retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome, and a broad range of other conditions. The study, due to begin recruitment in December, will help clinical researchers gain a better understanding of the course of retinal degeneration and vision loss for people with mutated genes that have not been well-characterized in the clinic. The study will also help the IRD research and therapeutic development community: 1) identify more people for clinical trials of therapies, 2) inform design of clinical trials, and 3) identify therapeutic targets for more people. "A majority of the 300 genes linked to inherited retinal diseases have not been well characterized in the clinic. That is, we do not have a good understanding of retinal degeneration and disease progression for those affected by these mutated genes," says Todd Durham, PhD, senior vice president, clinical development, at the Foundation Fighting Blindness. "We are excited to launch a highly inclusive study to benefit a large segment of the population affected by IRDs, which are so genetically diverse." "We are at a promising juncture in the clinical development of genetic therapies for inherited retinal diseases," says José-Alain Sahel, MD , distinguished professor and chairman of the department of ophthalmology at the University of Pittsburgh School of Medicine and Uni-Rare Study chair. "With dozens of emerging treatments in, or moving toward, clinical trials, the Uni-Rare Study will be invaluable in defining and validating outcome measures for clinical trials and establishing the infrastructure for multicenter studies. The Foundation Fighting Blindness, with its strong presence and relationships in the global IRD research community, is the ideal partner to lead the effort." The two-part Uni-Rare Study will be conducted by the Foundation's Clinical Consortium, a global, 40-site network of clinical research centers equipped with the experts and resources needed to launch IRD clinical trials and natural history studies. The Jaeb Center for Health Research will serve as the coordinating center. In the first part of the study, all participants will have an initial evaluation to collect their genetic data and clinical measures. When the study is initiated, study participants whose IRD is caused by variants in the RDH12 and MYO7A genes may qualify for the second part of the study, which involves collecting clinical measures for an additional four years. Additional gene cohorts for this part of the study are expected to be added in the future. The Foundation has received support for the study from families affected by Usher syndrome type 1B, Save Sight Now, Opus Genetics, Atsena Therapeutics, and Cove Therapeutics and is seeking additional support to add further gene cohorts to the second part of the study. "Opus is pleased to be an early supporter of this first-of-its-kind in ophthalmology initiative to understand natural histories around rare diseases of the retina in order to accelerate research and therapeutic development for these blinding conditions," said Jennifer Hunt, chief development officer at Opus Genetics. "Atsena Therapeutics is pleased to support the Uni-Rare natural history study and the inherited retinal disease community as we continue advancing the preclinical development of our dual AAV vector-based gene therapy to prevent blindness from USH1B," said Kenji Fujita, MD, chief medical officer at Atsena Therapeutics. "We are proud to sponsor such a robust interrogation of natural history that will be used to inform future clinical development plans and improve patient outcomes; these data will be fundamental for our understanding of several diseases and will serve to accelerate the development of Cove's non-viral gene therapies," said Niren Shah, PharmD, MBA, CEO and co-founder of Cove Therapeutics. IRD patients and physicians with IRD patients can get more information at clinicaltrials.gov. Patients with IRDs caused by mutations in the following genes are not eligible for the study: ABCA4, CEP290, CHM, CNGA3, CNGB3, EYS, GUCY2D, PCDH15, PROM1, RHO, RPE65, RPGR, USH2A. These genes are or have been part of other natural history studies or clinical trials. About the Foundation Fighting Blindness Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised more than $891 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration, and Usher syndrome. Visit FightingBlindness.org for more information. About Opus Genetics Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness's venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit opusgenetics.com. About Atsena Therapeutics Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company's ongoing Phase I/II clinical trial is evaluating a potential therapy for a form of LCA, one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina's Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. About Cove Therapeutics Cove Therapeutics is a privately held biotechnology company focused on developing non-viral, non-lipid, nanoparticle gene therapies for orphan and chronic prevalent indications. The Company utilizes a nanoparticle technology that overcomes limitations in gene therapy by enabling cell-specific tropism, scalable manufacturing, redosing, and packaging of large genes while maintaining a favorable safety profile. Together with these nanoparticles, Cove's lead franchise for ophthalmic conditions leverages a novel outpatient suprachoroidal device that enables broad distribution of therapeutics. Learn more about the Company through direct contact — Stephen Jasper, Gilmartin Group, [email protected], 858-525-2047. Media Contact: Chris Adams Vice President, Marketing & Communications [email protected] 410-423-0585 SOURCE Foundation Fighting Blindness